EU Regulation Is Hampering My Cancer Research

If my patient Jimmy had been diagnosed with cancer back in the
1980s, his prognosis would have been grim. Jimmy is eight months
old and has hepatoblastoma, a rare type of liver cancer; in the
past the only hope of cure was surgery (if possible), together
with old, ineffective chemotherapy treatments.

However, new drugs, tested in a series of clinical trials
throughout Europe involving more than 500 children, have
dramatically boosted survival rates in less than 10 years.
Jimmy’s chances of recovery are now 75 per cent, up from 30 per
cent.

As a consultant who has treated children with cancer for more
than two decades, I have seen the difference trials have made.
Every year, 1,500 children are diagnosed with cancer in Britain.
Forty years ago, a quarter were expected to survive – now four in
five will do so, as my co-authors and I reported in the Annals of
Oncology last week. But my concern is that unwieldy EU regulation
is now threatening the advances we have made – and future
progress.

A clinical trial compares current treatment with a modification
of that treatment or with a new one that doctors and scientists
hope will work better. Survival rates can be boosted – as we see
in cases like Jimmy’s. It also means that we can develop
treatment regimes with less severe side effects.

I’ve just completed a 10-year international clinical trial into
Wilms’ tumour (a kidney cancer), in which more than 4,000
children took part. One of the drugs used to treat it can cause
heart failure in later life. We’ve been able to show that an
additional 25 per cent of children with Wilms’ tumour don’t need
this drug. We also discovered a new sub-type of Wilms’ tumour
that benefits from stronger treatment; we are now doing more
molecular tests to see how many sub-types of tumour there are so
that we can personalise each child’s treatment further.

But the 2004 EU Clinical Trials Directive is putting such
breakthroughs at risk. It was brought in with the laudable aim of
harmonising trial methods and setting standards for
administration. The impact is different; the number of new
clinical trials in Europe between 2007 and 2011 dropped by a
quarter.

The reasons? First, the directive applies the same standards of
safety reporting to drugs that have been used for decades as
drugs never used in humans before. So every time a child enrolled
in a trial is admitted to hospital – even if it is nothing to do
with the trial – this must be reported, generating a huge amount
of paperwork.

The directive has also made it mandatory for each country to
ensure participants get anti-cancer drugs free of charge. In
Britain, the NHS provides standard drugs for free, and we work
with drug companies to provide new drugs for children’s cancer
trials. But in Poland or Hungary, for example, the institution
leading the trial is expected to pay treatment costs, which most
cannot afford.

Added to this is the EU’s insistence on insurance. In Britain we
have NHS indemnity insurance but in other countries the cost has
gone up 100-fold.

Bureaucracy has slowed everything down. Yet pharmaceutical
companies with drugs to trial want partnerships with institutions
that can get a study running in 60 days. We’re a long way off
that. So instead such companies are going to North America, China
and India. That means children in Britain may not get new drugs
for 10 years or more because we won’t have early access to
information that shows which treatments are working.

I wholeheartedly support current efforts to reform the directive.
We need to ensure a more proportionate approach to risk, more
flexibility and less paperwork. Most of all, drugs that are in
habitual use should no longer be subject to the full force of
regulation.

If this doesn’t happen, we’ll be stuck using standard treatments
rather than finding new ones. We’ll see too many children with
rarer cancers continuing to die, and others enduring serious side
effects such as hearing problems and infertility.

I’m not sure the trial I’ve just completed would have gone ahead
if the directive had been in place a few years earlier. It
certainly would have taken much longer to set up. But it’s
already making a big difference to many children with kidney
cancer – and has given us vital information for the future.

Kathy Pritchard-Jones is professor of paediatric oncology at
Great Ormond Street Hospital and the University College London
Institute of Child Health